JERRY LEWIS MDA LABOR DAY TELETHON
WILL FEATURE RESEARCH ADVANCES
IN 2007- 2008

TUCSON, Ariz., Aug. 4, 2008 — New drugs developed with support from by the Muscular Dystrophy Association in collaboration with biotechnology companies, new uses for existing medications, and new strategies to insert or modify genes are among the many achievements of MDA’s biomedical research program for muscular dystrophy and related diseases that will be featured on the 43rd annual Jerry Lewis MDA Labor Day Telethon.

The show will be broadcast to nearly 40 million viewers in the United States and Canada by some 190 television stations comprising MDA’s “Love Network.” Millions more worldwide will be able to see the Telethon on the Internet via RealNetworks at mda.org.

Jerry Lewis, legendary entertainer and MDA’s National Chairman, once again will host the show, with help from a collection of top-notch talent. In addition to the wide variety of entertainment for which it is known, the 21 1/2 -hour extravaganza will feature research news and profiles of people affected by muscle-wasting conditions.

“Nothing can deter me from supporting MDA’s top-tier, dedicated researchers in their mission to cure muscular dystrophy,” Lewis said. “Not floods, hurricanes or high gas prices. Now more than ever, we have a real shot at curing some of the most devastating killers of children known to mankind.”

Among the more than 40 conditions covered by MDA are nine forms of muscular dystrophy, ALS (amyotrophic lateral sclerosis, or Lou Gehrig’s disease), spinal muscular atrophy (SMA) and Charcot-Marie-Tooth disease (CMT).

The 2007 Telethon was viewed by some 40 million people, and its final tote board figure was a record $63.8 million.

MDA dedicates 78.4 cents of every dollar it spends to research or services.

In addition to MDA research, which costs approximately $82 a minute, Telethon funds provide:

• direct medical care through MDA’s network of 225 clinics, of which 38 are designated MDA/ALS centers;
• accessible summer camps for children with any one of the diseases in MDA’s program;
• durable medical equipment, including communication devices;
• some 300 support groups; and
• a comprehensive educational program for patients and professionals.

Research Progress

This year, MDA has allocated about $38 million for research, funding some 333 projects worldwide. MDA-funded scientists are making progress in understanding disease mechanisms, as well as testing promising treatments.

• Research at the MDA-supported ALS Therapy Development Institute in Cambridge, Mass., has made tremendous progress in standardizing animal models of the disease used in experiments, and in tracking biochemical changes associated with the disease. MDA has pledged $18 million to ALS TDI over three years.
• The Translational Genomics Research Institute in Phoenix published an article in the New England Journal of Medicine about its findings of genetic differences between people with and without ALS – a cutting-edge project funded by MDA’s Augie’s Quest ALS research initiative. Small differences in various genes were identified, with one standing out as particularly significant. The function of that gene remains unknown.
• MDA began funding a trial of lithium carbonate, normally prescribed to treat bipolar disorder, in people with ALS, after a small trial in Italy showed promise for slowing progression of the disease.
• Further testing of the recently approved drug Myozyme, developed by Genzyme Corp. of Cambridge, Mass., with supplemental support from MDA, showed it improves endurance and pulmonary function in older children and adults with late-onset Pompe disease. Earlier tests showed it prolongs life in babies and young children with this metabolic muscle disease.
• The experimental medication PTC124, developed by PTC Therapeutics of South Plainfield, N.J., with MDA support, restored production of the needed protein dystrophin in six boys with a particular form of Duchenne muscular dystrophy (DMD), who took it at a high dose for a month. Earlier, about half of 26 boys with DMD who took PTC124 at a lower dose also began making dystrophin. The drug is designed to make muscle cells ignore an aberrant molecular “stop sign” in the dystrophin gene, a mutation that occurs in about 15 percent of DMD cases.
• A trial of an “exon skipping” compound called AVI-4658, developed by AVI BioPharma of Portland, Ore., opened to approximately nine boys with DMD in the United Kingdom. AVI-4658 is designed to make cells “skip over” erroneous DNA genetic instructions and create functional dystrophin protein, which is missing in DMD. MDA aided the development of this compound with support to scientists in Australia.
• Four boys with DMD who received muscle injections of the exon skipping compound PRO051 began making the needed protein dystrophin in their leg muscles. The drug was developed by the Dutch company Prosensa and by an MDA-supported scientist at Leiden (Netherlands) University.
• Six boys with DMD who received arm-muscle injections of miniaturized dystrophin genes inside viral transporters showed that the gene therapy compound can be safely tolerated. The compound, called Biostrophin, was developed by Asklepios Biopharma of Chapel, Hill, N.C., with substantial support from MDA.
• Plans advanced for a similar MDA-supported clinical trial to insert the alpha-sarcoglycan gene into people with a form of limb-girdle muscular dystrophy (LGMD).

Since last year’s Telethon, MDA-funded researchers have also:

• continued to study ways of counteracting the effects of myostatin, a protein that limits muscle growth;
• found that genes for the muscle protein utrophin benefited mice with a DMD-like disease;
• found that muscle stem cells taken from people with DMD and then genetically corrected led to a significant recovery of muscle function, as well as production of dystrophin, in dystrophin-deficient mice with a DMD-like disease;
• released, in conjunction with pulmonary specialists, recommendations for anesthesia usage and preoperative care for patients with DMD;
• found that boys with DMD who take the corticosteroid prednisone to preserve muscle strength seemed to benefit almost as much from a higher-dose/two-days-a-week schedule of the drug as they did from a moderate-dose/daily schedule, with fewer negative side effects on their growth;
• found the drug sildenafil (Viagra) significantly improved heart function in mice with a disease resembling DMD;
• discovered that nervous-system support cells known as astrocytes may play a larger role in ALS than previously thought;
• discovered that the barrier that normally exists between circulating blood and the spinal cord breaks early in mice with an ALS-like disease;
• continued to study genetic and environmental risk factors that may contribute to ALS development;
• developed guidelines for disease management in spinal muscular atrophy (SMA) in conjunction with other SMA groups;
• identified the gene for a rare form of SMA that stems from an error in an X-chromosome gene;
• continued a clinical trial of hydroxyurea, a drug that may increase the level of the needed SMN protein, in children with SMA;
• continued testing high-dose ascorbic acid (vitamin C) in people with one form of Charcot-Marie-Tooth disease (CMT), a peripheral nerve disorder;
• continued studying the DNA, muscle tissue and medical histories of people with myotubular myopathy (muscle disease), nemaline myopathy and undefined myopathies to further understand these disorders.

Comprehensive Services

• MDA is allocating approximately $92.5 million this year to health care and community services for people with neuromuscular diseases. With help from Telethon donations, MDA has:
• registered tens of thousands of visitors to 225 hospital-affiliated clinics, of which 38 are designated MDA/ALS centers, for diagnosis, follow-up treatment, and physical, occupational and respiratory therapy consultations;
• provided financial assistance with the purchase and repair of wheelchairs, leg braces and communication devices;
• loaned thousands of pieces of adaptive equipment, such as hydraulic lifts, wheelchairs and hospital beds;
• sent more than 4,000 kids to some 90 accessible MDA summer camps in the United States and Puerto Rico at no cost to their families;
• paid for flu vaccinations to help prevent potentially deadly respiratory complications in those with weakened respiratory muscles due to muscular dystrophy or other related diseases;
• facilitated some 300 support groups, where individuals and their families affected by neuromuscular disease can socialize and exchange information.

Professional and Public Health Education

Telethon donations help MDA fund scientific and medical conferences among researchers, physicians and other health care professionals.

Contributions also make possible the publication of informative booklets, care guides, videos, the award-winning bimonthly Quest magazine, the monthly MDA/ALS Newsmagazine, and educational booklets for those who have just received a diagnosis of a disease in MDA’s program.

MDA shares its message and provides up-to-date knowledge and news through public speaking engagements, media interviews, public service announcements, educational videos and four Web sites (including one in Spanish): www.mda.org, www.als-mda.org, www.augiesquest.org, and www.mdaenespanol.org.